Pleuropulmonary blastoma and DICER1-related tumor predisposition: from clinicopathologic observations to clinical trial.

IF 2.2 3区医学 Q2 PEDIATRICS

Current opinion in pediatrics Pub Date : 2025-02-01 Epub Date: 2024-12-06 DOI:10.1097/MOP.0000000000001431

Alexander T Nelson, Kenneth S Chen, Kris Ann P Schultz

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引用次数: 0

Abstract

Purpose of review: Pleuropulmonary blastoma (PPB) is a rare primary lung neoplasm of infancy and childhood. The purpose of this review is to highlight recent developments in our understanding of PPB and research strategies to facilitate future rare cancer research.

Recent findings: The International PPB/DICER1 Registry has recently assembled the largest-ever cohorts of type I and Ir PPB and type II and III PPB. These analyses were strengthened by robust histologic, genetic and longitudinal data made possible by systematic data collection and abstraction and dedicated central pathology review. These cohorts have laid the groundwork for a prospective consortium-based clinical trial to assess response to camptothecins in type II and III PPB and standardize the use of chemotherapy in type I PPB.

Summary: Significant strides in the study of PPB have been made through clinical, laboratory and translational research, multidisciplinary collaborations and the generous contributions of patients, families and referring physicians. Ongoing advancements will continue to depend on multidisciplinary, multiperspective global collaborations.

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胸膜肺母细胞瘤与dicer1相关的肿瘤易感性：从临床病理观察到临床试验

回顾目的：胸膜肺母细胞瘤（PPB）是一种罕见的婴幼儿原发性肺肿瘤。这篇综述的目的是强调我们对PPB的理解和研究策略的最新进展，以促进未来罕见癌症的研究。最近的发现：国际PPB/DICER1登记处最近汇集了有史以来最大的I型和Ir型PPB和II型和III型PPB队列。通过系统的数据收集和提取以及专门的中心病理审查，这些分析得到了强有力的组织学、遗传和纵向数据的加强。这些队列为一项前瞻性的以联合体为基础的临床试验奠定了基础，以评估喜树碱对II型和III型PPB的疗效，并规范I型PPB化疗的使用。摘要：通过临床、实验室和转化研究、多学科合作以及患者、家属和转诊医生的慷慨贡献，PPB的研究取得了重大进展。持续的进步将继续依赖于多学科、多视角的全球合作。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Current opinion in pediatrics 医学-小儿科

CiteScore

6.20

自引率

0.00%

发文量

184

审稿时长

6-12 weeks

期刊介绍： Current Opinion in Pediatrics is a reader-friendly resource which allows the reader to keep up-to-date with the most important advances in the pediatric field. Each issue of Current Opinion in Pediatrics contains three main sections delivering a diverse and comprehensive cover of all key issues related to pediatrics; including genetics, therapeutics and toxicology, adolescent medicine, neonatology and perinatology, and orthopedics. Unique to Current Opinion in Pediatrics is the office pediatrics section which appears in every issue and covers popular topics such as fever, immunization and ADHD. Current Opinion in Pediatrics is an indispensable journal for the busy clinician, researcher or student.